The potential cost-effectiveness of novel cord blood therapies in children with autism spectrum disorder.
The potential cost-effectiveness of novel cord blood therapies in children with autism spectrum disorder.
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DOI：
10.1371/journal.pone.0282906
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发表时间：
2023
期刊：
影响因子：
3.7
通讯作者：
中科院分区：
文献类型：
--
作者：
模拟自闭症谱系障碍（ASD）潜在脐带血治疗的长期临床和经济结局。使用寿命期间ASD的马尔可夫微观模拟来比较两种策略：1）护理标准（SOC），包括行为和教育干预，和2）除了SOC之外的新脐带血（CB）干预。反映行为结果的输入数据包括基线Vineland适应行为量表（VABS-3）、每月VABS-3变化和CB干预对基于随机、随机、随机和随机的适应行为的功效。安慰剂对照试验（DukeACT）。质量调整生命年（QIRES）与VABS-3相关。合并了ASD儿童（15，791美元，2-17岁）和ASD成人（56，559美元，18岁以上）以及CB干预（范围15，000 - 45，000美元）的费用。探讨了替代CB的疗效和成本。我们将模型预测的结果与已公布的预期寿命、平均VABS-3变化和终身成本数据进行了比较。SOC和CB策略中的未贴现生命周期QQL分别为40.75和40.91。SOC策略的贴现寿命成本为1，014，000美元，CB的范围为1，021，000 - 1，058，000美元，CB干预成本为8，000 - 45，000美元。在15，000美元的成本下，CB是临界成本效益（ICER = 105，000美元/QALY）。在单因素敏感性分析中，CB成本和疗效是影响CB ICER的最重要因素。CB干预在成本<15，000美元且功效≥2.0时具有成本效益。五年期医疗保健支付者预计15，000美元CB成本的预算支出为38.47亿美元。在某些情况下，旨在改善自闭症适应行为的适度有效干预可能具有成本效益。干预成本和效果对成本效果的影响最大，应针对性地提高经济效益。
To model the long-term clinical and economic outcomes of potential cord blood therapy in autism spectrum disorder (ASD). Markov microsimulation of ASD over the lifespan was used to compare two strategies: 1) standard of care (SOC), including behavioral and educational interventions, and 2) novel cord blood (CB) intervention in addition to SOC. Input data reflecting behavioral outcomes included baseline Vineland Adaptive Behavior Scale (VABS-3), monthly VABS-3 changes, and CB intervention efficacy on adaptive behavior based on a randomized, placebo-controlled trial (DukeACT). Quality-adjusted life-years (QALYs) were correlated to VABS-3. Costs for children with ASD ($15,791, ages 2–17 years) and adults with ASD ($56,559, ages 18+ years), and the CB intervention (range $15,000–45,000) were incorporated. Alternative CB efficacy and costs were explored. We compared model-projected results to published data on life-expectancy, mean VABS-3 changes, and lifetime costs. Undiscounted lifetime QALYs in the SOC and CB strategies were 40.75 and 40.91. Discounted lifetime costs in the SOC strategy were $1,014,000, and for CB ranged from $1,021,000-$1,058,000 with CB intervention cost ($8,000-$45,000). At $15,000 cost, CB was borderline cost-effective (ICER = $105,000/QALY). In one-way sensitivity analysis, CB cost and efficacy were the most influential parameters on CB ICER. CB intervention was cost-effective at costs<$15,000 and efficacies ≥2.0. Five-year healthcare payer projected budgetary outlays at a $15,000 CB cost were $3.847B. A modestly effective intervention designed to improve adaptive behavior in autism can be cost-effective under certain circumstances. Intervention cost and efficacy most affected the cost-effectiveness results and should be targeted to increase economic efficiency.
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DOI：
10.1177/1362361317716606
发表时间：
2018-11
期刊：
Autism : the international journal of research and practice
影响因子：
--
作者：
Duncan A;Ruble LA;Meinzen-Derr J;Thomas C;Stark LJ
通讯作者：
Stark LJ
影响因子：
5.1
作者：
Dawson, Geraldine;Sun, Jessica M.;Kurtzberg, Joanne
通讯作者：
Kurtzberg, Joanne
影响因子：
3.9
作者：
Meyer, Allison T.;Powell, Patrick S.;Klinger, Laura G.
通讯作者：
Klinger, Laura G.
影响因子：
8
作者：
Lavelle TA;Weinstein MC;Newhouse JP;Munir K;Kuhlthau KA;Prosser LA
通讯作者：
Prosser LA
影响因子：
7.2
作者：
Kaimal, Anjali J.;Smith, Catherine C.;Cheng, Yvonne W.
通讯作者：
Cheng, Yvonne W.